| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 2529795 | Current Opinion in Pharmacology | 2015 | 8 Pages |
•CAV-2 vectors preferentially transduce neurons in rodents and primates.•Retrograde axonal transport leads to widespread biodistribution in the brain parenchyma.•Helper-dependent CAV-2 vector treat global brain pathology in MPS brain.•Use of CAV-2 vector for fundamental neurobiology is going viral.
Canine adenovirus type 2 (CAV-2) vectors are powerful tools for fundamental and applied neurobiology due to their negligible immunogenicity, preferential transduction of neurons, widespread distribution via axonal transport, and duration of expression in the mammalian brain. CAV-2 vectors are internalized in neurons by the selective use of coxsackievirus and adenovirus receptor (CAR), which is located at the presynapse in neurons. Neuronal internalization and axonal transport is mediated by CAR, which potentiates vector biodistribution. The above characteristics, together with the ∼30 kb cloning capacity of helper-dependent (HD) CAV-2 vectors, optimized CAV-2 vector creation, production and purification, is expanding the therapeutic and fundamental options for CNS gene transfer.
